My expertise is in both oncology and hematology. Although the majority of my time is spent in the laboratory, I enjoy taking care of patients at Children's Hospital. My research has given me a broad clinical approach that helps me diagnose and treat the rare child who is "off the grid," particularly when there is an oncology/hematology problem that has gone unsolved. I also specialize in hematopoietic stem cell transplant (bone marrow transplant) and use my expertise in transplant to help treat patients with autoimmune diseases.
My clinical research is focused on better understanding diseases of white blood cells in children, in particular leukemia and a rare disorder called autoimmune lymphoproliferative syndrome (ALPS). I focus on creating novel therapeutics for leukemia and ALPS, and have been successful in bringing drugs from the bench to the bedside to treat children with these conditions.
I am the principal investigator for a clinical trial that is using the mTOR inhibitor rapamycin (sirolimus) in children with multiple medical conditions. mTOR is a key protein in a pathway that regulates cell activities including growth and survival. The autoimmune conditions we are looking at in this trial include ALPS, chronic Immune Thrombocytopenic Purpura (ITP, a blood disorder that involves the immune and blood clotting systems) and lupus (a chronic, inflammatory autoimmune disorder.) I also run an ongoing multi-institutional trial to diagnose and better characterize children with ALPS.
My basic science research focuses on the same two areas: leukemia and autoimmune diseases. My first area of interest is the signal transduction pathways and inhibitors of those pathways in acute lymphoblastic leukemia (ALL). My lab targets the mTOR signaling pathway with novel therapeutics. We use xenograft (tissue grafts from one species to another) models to study the efficacy and action of those therapeutic agents directly on leukemia cells removed from children. This work has led to a number of nationwide Children's Oncology Group-initiated clinical trials that test mTOR inhibitors in patients with ALL.
The other major area of basic research in my lab centers on investigating novel therapeutics and signaling pathways in autoimmune diseases, particularly ALPS. Using preclinical models, I am targeting both mTOR and Notch cell signaling as treatment approaches for patients with ALPS and other autoimmune diseases. I am also interested in understanding lymphocyte biology in ALPS and other autoimmune diseases.
I became interested in ALPS as a doctor in training. We had patients with this disorder and couldn't figure out what it was or how to treat it. Now I run the one of the biggest clinics in the world for ALPS. Because of our new treatments, patients are doing better than ever before.
At Children's Hospital, we are somewhat unique in that we value and excel at both patient care and basic science and clinical science. We are able to put our full weight on all the pathways to help children with cancer and blood disorders.
« Back to Previous Page